Metabolic Control and Frequency of Clinical Monitoring Among Canadian Children With Phenylalanine Hydroxylase Deficiency: A Retrospective Cohort Study.

Achieving and maintaining metabolic control is critical for children with phenylalanine hydroxylase (PAH) deficiency. This retrospective longitudinal cohort study investigated metabolic control and monitoring frequency of children with PAH deficiency (≤ 12 years) treated at one of 12 pediatric metabolic centres across Canada. We abstracted data from medical charts and analyzed outcomes by age and diagnostic classification, using mixed effects regression.

Development of a protocol complexity tool: a framework designed to stimulate discussion and simplify study design.

Background: Over the last decade, the pharmaceutical industry has witnessed longer, more complex, and expensive clinical trials. This complexity contributes to delays in clinical trial implementation, execution, monitoring, recruitment, data cleaning, and interpretation. Our aim was to develop a protocol complexity tool (PCT) to simplify clinical trial execution without compromising science or quality.

Analysis of Inositol Phosphates, Diphosphoinositol Phosphates, and Their Analogs by HPLC on an Acid-Compatible Stationary Phase with Detection by Complexation with Ferric Ion.

High-performance anion-exchange chromatography with acid eluents allows for effective separation of myo-inositol tetrakisphosphates, myo-inositol pentakisphosphates, diphospho-myo-inositol phosphates (inositol pyrophosphates), inositol hexakisphosphates, inositol pyrophosphate analogs, and carbohydrate-based analogs of inositol phosphates. All are detectable by postcolumn addition of ferric ion. In the following, we provide examples drawn from characterization of ITPK, IPK1, and IP 3K enzymes.